TACRINE HYDROCHLORIDE TREATMENT IND - METHODS FOR RAPID PHYSICIAN ANDPATIENT ENROLLMENT AND DATA-RETRIEVAL

A Treatment IND (TIND) is a mechanism available to the Food and Drug A dministration (FDA) in the United States by which promising new drugs can be provided to patients with life-threatening illnesses. In many i nstances, the illness is life-threatening but of relatively low incide nce, making the demand for the new treatment limited. However, if the disease is more prevalent and incidence is increasing, the demand for access to an experimental therapy may be substantially greater. Novel approaches and technologies would help manage recruitment of physician s, enrolment of patients and retrieval and timely analysis of data. Su ch was the case in the TIND for tacrine hydrochloride (Cognex(R)), a c holinesterase inhibitor which was under development for the treatment of patients with Alzheimer's Disease (AD), There were an estimated 4 m illion prevalent cases of AD in the US for which no approved therapeut ic option was available at the time this TIND was initiated. We antici pated that there could be a large demand by both physicians and patien ts to enrol in the TIND. Therefore, to meet this demand, various mecha nisms were employed to allow rapid enrolment and drug shipments to the patient. In addition, physicians who participated in the TIND were ab le to use a telephone touch-tone data entry system for reporting data and ordering new supplies of tacrine for their patients. Serious adver se events were reported directly to trained operators and summarized o n a weekly basis for reporting to the FDA. At the time the programme w as terminated, nearly 2000 physicians had enrolled to participate in t he TIND and nearly 10,000 patients had received tacrine under the prog ramme. The methods employed in this study to collect clinic visit and safety data met both regulatory and good clinical practice guidelines. In summary, a large volume of data was handled rapidly and efficientl y in this programme. (C) 1997 John Wiley & Sons, Ltd.